FDA Grants Accelerated Approval to Yuviwel (navepegritide) for Children with Achondroplasia

Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has granted approval under the FDA’s Accelerated Approval Program for Yuviwel (navepegritide; developed as TransCon® CNP), the first and only once-weekly treatment indicated to increase linear growth in children 2 years of age and older with achondroplasia with open epiphyses and the only one to provide continuous systemic exposure to CNP over the weekly dosing interval. Continued approval for this indication, which was based on an improvement in annualized growth velocity (AGV), may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

• The first and only approved achondroplasia therapy to provide continuous systemic exposure to CNP over the weekly dosing interval
• Commercial availability expected during early part of Q2 2026
• Rare Pediatric Disease Priority Review Voucher granted in connection with approval

Achondroplasia is a rare genetic condition causing skeletal dysplasia and, for many affected individuals, an increased risk of muscular, neurological, and cardiorespiratory complications. Yuviwel is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia.

“The approval of once-weekly Yuviwel is a major step forward in the treatment of children with achondroplasia, giving physicians for the first time the option of prescribing a once-weekly medicine backed by compelling efficacy and excellent tolerability data from three randomized, double-blind, placebo-controlled clinical trials,” said Carlos A. Bacino, MD, FACMG, Professor of Molecular and Human Genetics, Baylor College of Medicine and Texas Children’s Hospital. “My goal is to help children and parents develop care plans tailored to their individual needs and objectives, and I look forward to adding Yuviwel to my discussions with them.”

“Little People of America, the largest national advocacy and support organization for people with dwarfism, is committed to ensuring that the voices of people with dwarfism remain central in conversations about research and medical options such as Yuviwel,” said the Board of Directors of Little People of America. “We champion dwarf and disability pride, advocate for inclusion and respect, and foster open dialogue across diverse perspectives. Our goal is to empower individuals and families to make healthcare decisions that reflect their own values and experiences, while pushing for research efforts and new treatment options such as this that could have the potential to support outcomes that truly matter to our community.”

The FDA based its approval of Yuviwel on their review of the clinical package for TransCon CNP submitted with the Company’s New Drug Application, which included safety and efficacy data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data. The pivotal ApproaCH Trial data is available in JAMA Pediatrics.i

“We are confident in Yuviwel’s potential to transform the treatment of achondroplasia and are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. “We have listened to advocacy groups for people with dwarfism to ensure we address what the community actually cares about. This reflects our ongoing commitment to pursue outcomes that patient communities have told us are important to them, and gives the achondroplasia community a new way to look at the promise of pharmacological treatment options.”

Ascendis expects to make Yuviwel available through prescribing physicians in the United States during the early part of the second quarter of 2026. Ascendis plans to offer a suite of patient services for Yuviwel through its U.S. Ascendis Signature Access Program (A.S.A.P.), including support navigating the treatment journey and financial assistance programs for eligible patients.

With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV), which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. This program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.

FDA Grants Accelerated Approval to Yuviwel (navepegritide) for Children with Achondroplasia

FDA Approves Voxzogo (vosoritide) to Increase Linear Growth in Children with Achondroplasia

 

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN)  announced  the U.S. Food and Drug Administration (FDA)  approval of VOXZOGO™ (vosoritide) for Injection, indicated to increase linear growth in pediatric patients with achondroplasia five years of age and older with open epiphyses (growth plates). This indication is approved under accelerated approval based on an improvement in annualized growth velocity (AGV). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory studies. To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.

Voxzogo is the first FDA approved treatment for children with achondroplasia. In patients with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 gene (FGFR3). Voxzogo, a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

"Voxzogo is a medical first that is rooted in BioMarin's focus on molecular genetics and targets the underlying cause of the condition. More than a decade of scientific research underpins the medical advance that Voxzogo represents. We thank the FDA for recognizing its value as the first therapeutic treatment option for children with achondroplasia," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We extend our gratitude to the community, clinical investigators and the children and their families, who participated and continue to participate in our comprehensive clinical research program as we continue to investigate the full potential of vosoritide."

"Achondroplasia is a lifelong genetic condition resulting from the disordered skeletal architecture caused by impaired endochondral bone growth throughout childhood," said Lynda Polgreen, M.D., an investigator in clinical trials for Voxzogo and an Investigator at The Lundquist Institute at Harbor-UCLA Associate Professor at David Geffen School of Medicine – UCLA. "This approval is an important milestone representing the first time that physicians will be able to offer a therapy targeted at the root cause of the condition for families of children with achondroplasia aged five and older."

"We applaud the FDA for recognizing the urgent unmet medical need for this progressive condition. As a parent of a child with achondroplasia, I see the availability of treatments that impact bone growth as an important step forward," said Amer Haider Co-Founder of Growing Stronger, an organization with a mission to improve the quality of medical care for little people through supporting research. The organization raises nonprofit donations that are granted to researchers focused on dwarfism.

Mr. Haider added, "BioMarin continues to support the achondroplasia community and has a long track record of advancing the standard of care in rare genetic conditions."

With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV), which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. The rare pediatric disease PRV program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.

The approval was based on the outcomes of a global randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of Voxzogo and the open-label extension of this Phase 3 study. The study enrolled 121 children aged 5 to 14.9 with achondroplasia. Baseline mean AGV in the placebo and Voxzogo groups was 4.06 cm/year and 4.26 cm/year, respectively. At week 52, the change from baseline in AGV was -0.17 cm/year for the placebo treated patients and 1.40 cm/year for the Voxzogo treated patients, resulting in a statistically significant improvement in AGV of 1.57 cm/year in favor of Voxzogo. After the 52 week double blind, placebo–controlled, phase 3 study, 58 subjects initially randomized to Voxzogo enrolled into an open–label extension. Among the subjects who had two years of follow–up since randomization, the improvement in AGV was maintained.

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